PHILADELPHIA, Nov. 10, 2020 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system disorders, today reported financial results for the third quarter ended September 30, 2020 and provided recent business highlights.

“Over the past quarter, we have made important strides in strengthening our operations to support the initiation of three Phase 1/2 clinical trials for our lead programs in infantile GM1, FTD-GRN, and Krabbe disease in the first half of 2021,” said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio. “We are particularly pleased with our progress in terms of patient identification, clinical trial site preparedness and manufacturing readiness. We also remain confident that we will receive FDA clearance for our investigational new drug application for PBGM01 for the treatment of infantile GM1. This has all been made possible because of the highly experienced, talented team that we have assembled over the past year to be ready to execute seamlessly in 2021 on our path to providing life-transforming gene therapies for patients with rare, monogenic CNS disorders.”

Recent Corporate Highlights

• Work progresses in anticipation of U.S. Food and Drug Administration (FDA) clearance for investigational new drug (IND) application for PBGM01: In August 2020, Passage Bio received its official letter from FDA confirming that the clinical hold of its IND for PBGM01 for the treatment of infantile GM1 gangliosidosis (GM1) was due solely to questions concerning the biocompatibility of the proposed intra cisterna magna (ICM) delivery device. Passage Bio is working diligently to resolve the clinical hold and, based on feedback from FDA, is conducting biocompatibility risk assessments and testing of the ICM device. The company remains confident that the IND will be cleared. As a result of these efforts to obtain FDA clearance of the device, Passage Bio now plans to initiate its Phase 1/2 trial in the first quarter of 2021 and to report initial 30-day safety and biomarker data mid-year 2021.
• Dedicated GMP manufacturing suite is complete: Construction and qualification of Passage Bio’s dedicated suite at Catalent are complete. With this suite, the company will now be able to provide clinical supplies for its lead pipeline products through early commercialization and expects to initiate manufacturing activities in the coming months. Clinical supply for the GM1 Phase 1/2 trial is already in place through Passage Bio’s ongoing partnership with Catalent.
• Orphan Drug Designation (ODD) granted by European Commission (EC) for PBGM01 – In October 2020, the EC granted ODD to PBGM01 for the treatment of GM1, a rare and often life-threatening monogenic recessive lysosomal storage disease caused by mutations in the GLB1 gene, resulting in rapidly progressing neurodegeneration. PBGM01 has previously been granted ODD and RPDD by FDA for the treatment of GM1.
• Rare Pediatric Disease Designation (RPDD) and ODD granted by FDA for PBKR03 – In October 2020, FDA granted RPDD and ODD to PBKR03 for the treatment of Krabbe disease, a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene resulting in progressive damage to both the brain and peripheral nervous system.
• Collaboration with Invitae in place to facilitate genetic testing and support diagnosis of patients with GM1 – Passage Bio recently announced it has entered into a collaboration with Invitae, a leading medical genetic testing company, to offer free genetic testing and counselling for GM1 through Invitae’s Detect Lysosomal Storage Disorders program to encourage early diagnosis. As part of the collaboration, Invitae will also provide educational information to patients and clinicians regarding clinical trials.
• Recently published preclinical data in peer-reviewed journals support advancement into the clinic of PBGM01 and PBFT02 – Passage Bio earlier announced the publications of preclinical data from the University of Pennsylvania’s Gene Therapy Program supporting the potential of PBGM01 and PBFT02 to correct the underlying genetic defect associated with GM1 and frontotemporal dementia (FTD) with a granulin (GRN) mutation, respectively. Data from a mouse model of GM1 demonstrated that a single intracerebroventricular injection of an optimized adeno-associated virus (AAV) into the cerebral spinal fluid (CSF) resulted in significant expression of ?-gal in the brain and peripheral tissues, and demonstrated dose-related reductions in neuronal lysosomal storage lesions, reduced neurological impairment and improvement in survival. Data from the FTD mouse model showed a single administration of an AAV containing the GRN gene resulted in elevated levels of progranulin in the brain and CSF, reduced lysosomal storage lesions, normalized lysosomal enzyme expression and corrected microgliosis. These GM1 data were published in Human Gene Therapy, and FTD-GRN data were published in Annals of Clinical and Translational Neurology.

Anticipated Upcoming Milestones

• Initiate Phase 1/2 trial for the Company’s lead program, PBGM01, for the treatment of patients with infantile GM1 in the first quarter of 2021. Report initial 30-day safety and biomarker data mid-year 2021.
• Continue to advance lead programs PBFT02 for the treatment of FTD-GRN and PBKR03 for the treatment of Krabbe disease toward Phase 1/2 clinical trial initiations in the first half of 2021.
• Continue to advance pre-clinical programs for PBML04 (Metachromatic leukodystrophy), PBLA05 (Amyotrophic lateral sclerosis) and PBCM06 (Charcot-Marie-Tooth Disease Type 2A).

Third Quarter 2020 Financial Results

• Cash Position:Cash, cash equivalents and marketable securities were $335.7 million as of September 30, 2020 as compared to $158.9 million as of December 31, 2019.
• Research and Development (R&D) Expenses:R&D expenses were $20.8 million for the quarter ended September 30, 2020, compared to $10.4 million for the same quarter in 2019.
• General and Administrative (G&A) Expenses: G&A expenses were $7.8 million for the quarter ended September 30, 2020, compared to $1.2 million for the same quarter in 2019.
• Net Loss: Net loss was $28.5 million, or a net loss of $0.63 per basic and diluted share, for the quarter ended September 30, 2020, compared to $11.4 million, or a net loss of $2.68 per basic and diluted share, for the same quarter in 2019.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvania’s Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with unparalleled access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at www.passagebio.com.